Small Molecules Directed at RNA
that Target 3D Structure

Case Number CH-039

Inventors: Elizabeth C. Theil, PhD

Abstract
The invention provides small molecules that bind to the novel features in RNA, leading to variation in the expression.  For example, compounds that bind to the ferritin mRNA block binding of regulatory proteins producing an increase in free translatable ferritin message.  Such an effect may lead to increased levels of ferritin protein and increased iron storage.

Most small molecules target proteins, and those that are directed at RNA, are targeted to two-dimensional structural features.  Lead compounds need to undergo optimization in order to improve selectivity.

Advantage
The invention is related to that described in US patent application number 5,834,199 (Case No. CH-016), by the same inventor, except that this new technology does not require transition metals, which makes these compounds more amenable to typical pharmaceutical methods.

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